Glucoimidazole and polyhydroxycyclohexenyl amine derivatives to treat gaucher disease
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United States of America Patent
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Issued Date -
Jun 23, 2005
app pub date -
Nov 12, 2004
filing date -
Nov 12, 2003
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Abstract
The present invention provides glucoimidazole (GIZ) and polyhydroxycyclohexenyl amine (PHCA) derivatives, methods of making them, and methods of use where the GIZ and PHCA derivatives have a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. More particularly, the present invention further provides a method for treating individuals having Gaucher disease by administering the novel GIZ or PHCA derivatives as 'active-site specific chaperones' for the mutant glucocerebrosidase associated with the disease.
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